New Drug PP188 Helps Sickle Cell Disease

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Sickle cell disease is an inherited blood disorder that affects red blood cells. People with sickle cell disease have red blood cells that contain an abnormal type of hemoglobin. Sometimes these red blood cells become sickle-shaped and have difficulty passing through small blood vessels. When sickle-shaped cells clump together and obstruct blood vessels, the result is often pain severe enough to require hospital admission.

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Researchers from the University of North Carolina at Chapel Hill studied more than 250 patients with sickle cell disease. Half of the patients received a drug known as purified poloxamer 188 (PP188), while the other half received a placebo. PP188 is a drug that improves blood flow by lowering blood flow resistance. Researchers found patients who received PP188 had a 6.4 percent reduction in duration of painful episodes compared with those who received a placebo.

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Researchers note the drug showed more promise in children younger than 15 years old. In those under 15, the painful episodes were reduced from an average of 6.2 days to 5.3 days and symptoms were more often resolved within a week in those who received PP188.

Researchers realize the reduction in painful symptoms was small. Nevertheless, researchers say a new option in the treatment of sickle cell disease can reduce the duration of painful of symptoms associated with the disease.

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